We're addressing perhaps the most difficult decision n-Lorem has to make in our work, declining a patient application. Today, our acceptance rate is ~59%. These decisions are made with heavy hearts at n-Lorem, and we are aware of how heartbreaking the decision to decline an application may be for a patient and their family. In this episode, we'll discuss the main reasons a patient's application may be denied, including safety, geography, and other factors.

On This Episode We Discuss:

• 1:43 – Non-technical issues: Outside of USA, inability to find treating physician, patient population size, if the patient is being treated by another experimental medicine, including repurposed drugs

• 7:15 – Definitions to better understand the conversation: Heterozygous mutation, haploinsufficiency

• 9:40 – n-Lorem must understand the cause of the disease, the nature of the mutation, and n-Lorem must be confident that the ASO designed would deliver value to the patient

• 12:35 – n-Lorem must be confident that only a single gene is causing phenotypes, and that the target is not a null mutation

• 13:37 – n-Lorem may accept patients, but then discontinue a program if the patient cells are unable to be grown for lab use and testing

• 14:56 – For patients requiring an allele-selective ASO, n-Lorem must identify non-pathogenic mutations to attempt to create an ASO capable of helping the patient

Links:

Donate - https://www.nlorem.org/donate/

Sponsors:

Hongene - https://www.hongene.com/

GondolaBio - https://gondolabio.com/